From the Guidelines
Primary biliary cholangitis (PBC) is best treated with ursodeoxycholic acid (UDCA) at a dose of 13-15 mg/kg/day, which should be started immediately upon diagnosis, as recommended by the most recent guidelines 1. This medication helps slow disease progression by reducing bile acid toxicity and improving bile flow. UDCA therapy should be continued indefinitely as it significantly reduces the risk of liver transplantation and improves survival when started early. The symptoms of PBC include:
- Fatigue
- Pruritus (itching)
- Jaundice
- Dark urine
- Pale stools
- Weight loss
- Abdominal pain
For patients who don't respond adequately to UDCA after 12 months (about 40% of patients), obeticholic acid (OCA) at 5-10 mg daily can be added as second-line therapy, as suggested by recent studies 1. Regular monitoring is essential, including:
- Liver function tests every 3-6 months
- Annual ultrasound surveillance for hepatocellular carcinoma in those with cirrhosis Symptom management is also important, with:
- Cholestyramine for pruritus
- Calcium/vitamin D supplementation to prevent metabolic bone disease PBC is an autoimmune disease that primarily affects middle-aged women, causing progressive destruction of small bile ducts, which leads to cholestasis, fibrosis, and potentially cirrhosis if left untreated. According to the latest guidelines, UDCA should be continued during pregnancy in patients with PBC, as it is safe in pregnancy and breastfeeding 1.
From the FDA Drug Label
OCALIVA can cause serious side effects including: Worsening of liver problems or liver failure, in some cases leading to liver transplant or death, has happened in people with primary biliary cholangitis (PBC) with liver cirrhosis when taking OCALIVA provider right away if you have any of the following symptoms of worsening liver problems during treatment with OCALIVA: swelling of your stomach-area from a build-up of fluid yellowing of your skin or the whites of your eyes black, tarry, or bloody stools coughing up or vomiting blood, or your vomit looks like "coffee grounds" mental changes such as confusion, sleepier than usual or harder to wake up, slurred speech, mood swings, or changes in personality Tell your healthcare provider right away if you have any of the following symptoms during treatment with OCALIVA and they are severe or do not go away: stomach-area pain nausea, vomiting, or diarrhea loss of appetite or weight loss new or worsening fatigue weakness fever and chills light-headedness less frequent urination
The symptoms of Primary Biliary Cholangitis (PBC) that may be experienced by patients taking OCALIVA include:
- Swelling of the stomach-area from a build-up of fluid
- Yellowing of the skin or the whites of the eyes
- Black, tarry, or bloody stools
- Coughing up or vomiting blood
- Mental changes such as confusion, sleepier than usual or harder to wake up, slurred speech, mood swings, or changes in personality
- Stomach-area pain
- Nausea, vomiting, or diarrhea
- Loss of appetite or weight loss
- New or worsening fatigue
- Weakness
- Fever and chills
- Light-headedness
- Less frequent urination
Treatment options for PBC include:
- OCALIVA, which can be used in combination with ursodeoxycholic acid (UDCA) or as monotherapy in patients unable to tolerate UDCA 2
- Ursodeoxycholic acid (UDCA) alone or in combination with OCALIVA
- Management of severe pruritus, which may include the addition of bile acid binding resins or antihistamines, OCALIVA dosage reduction and/or temporary dosing interruption 2
- Monitoring for changes in serum lipid levels during treatment with OCALIVA 2
From the Research
Symptoms of Primary Biliary Cholangitis (PBC)
- Pruritus (itching)
- Fatigue
Treatment Options for PBC
- First-line treatment: Ursodeoxycholic acid (UDCA) 3, 4, 5, 6, 7
- Second-line treatment: Obeticholic acid (OCA) for patients who do not respond to UDCA 4, 5, 6, 7
- Other options: Bezafibrate, budesonide, triple therapy with UDCA, OCA, and bezafibrate 4, 5
- Liver transplantation for refractory cases progressing to cirrhosis 5, 7
Evaluation of Treatment Response
- ALP, AST, and total bilirubin (TBIL) threshold values can be used to predict treatment outcomes 3
- A new criterion, Xi'an criterion, has been established to evaluate UDCA response at 1 month: ALP ≤ 2.5 × upper limit of normal (ULN), AST ≤ 2 × ULN, and TBIL ≤ 1 × ULN 3
- Patients who do not meet the defined criteria of response may require adjunctive therapy 7